Now play­ing catch-up to blue­bird, Ver­tex and CRISPR send in their pitch for blood dis­or­der cell ther­a­py

If all things go ac­cord­ing to plan, the FDA could start re­view­ing the first-ever fil­ing for a CRISPR ther­a­py this No­vem­ber.

Ver­tex and CRISPR Ther­a­peu­tics say they have wrapped up their lengthy talks with the agency and got­ten reg­u­la­tors on board for a rolling re­view be­gin­ning lat­er this year — with the sub­mis­sion pack­age due to be com­plete in the first quar­ter of 2023.

While the part­ners once hoped that they would be the first to bring a once-and-done ther­a­py to the US mar­ket for sick­le cell dis­ease (SCD) and trans­fu­sion-de­pen­dent be­ta tha­lassemia (TDT), blue­bird bio beat them to the punch with the re­cent FDA ap­proval of Zyn­te­glo. The fil­ing al­so marks a slight de­lay to ear­li­er plans of fil­ing in late 2022, al­though it wasn’t en­tire­ly un­ex­pect­ed.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.